Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

NCT05105685 | Completed Phase 1 DMC

• 1 other identifier: 395779225
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

In summary, this piot study with 6 participants shown that recombinant human growth hormone (rhGH) has a positive effect on the treatment with PMS. In addition, This study indicated that rhGH can improve PMS symptoms via increase the level of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3). RhGH may be low cost, more accessible, alternative treatment for PMS.

Conditions

Phelan-McDermid Syndrome; Growth Hormone Treatment

Keywords

Phelan-McDermid Syndrome; Growth Hormone Treatment; cross-over; Placebo-controlled

Outcome Measures

Primary Outcomes (4)

• level of serum IGF-1 and IGFBP-3

  All laboratory indices were completed in the morning while the participants fasted and were analysed by the laboratory of the investigators' hospital.

  3 months

• Chinese version of the Gesell Development Scale (GDS)

  To measure neuropsychological development. The development quotient (DQ) in the GDS was used to quantify neurodevelopment, which can indicate the level of neurodevelopment and is interpreted as follows: DQ=86 as normal, DQ 76 to 85 as marginally delayed, DQ at 55-75 as slightly delayed, DQ at 40 to 54 as moderately delayed, and DQ⩽39 as severely delayed.

  3 months

• Simplified Chinese version of the Aberrant Behavior Checklist (SC-ABC)

  Different from its English version that created by Krug in 1980 , SC-ABC was based on the study of Krug in 2009, in which proved ABC can be used in 14 months children, then it was translated into Simplified Chinese by the researchers of Peking University Sixth Hospital. To date, the SC-ABC scale has been verified and widely used for more than 10 years to assess the changes of symptoms in Chinese children aged from 14 months to 14 years old with behavioral problems. This checklist includes 57 items and five subscales: sensory behavior, social relating, body and object use, language and communication skills, and social and adaptive skills. Each item was scored from 0 to 3,with higher scores indicate more severe symptoms.

  3 months

• Adverse events

  Adverse events (AEs) were measured during the trial during via monitoring visits or phone calls using an adapted semi-structured interview every two weeks

  3 months

Study Arms (2)

Group A

PLACEBO COMPARATOR

Subjects were randomly divided into two different groups. Group A began with placebo while group B with rhGH. Randomisation of the treatment order (i.e., starting with rhGH or placebo) was done through a permuted four-block designed by the physician, who was the only one not blinded for treatment allocation.

Drug: Saline

Group B

EXPERIMENTAL

Subjects were randomly divided into two different groups. Group A began with placebo while group B with rhGH. Randomisation of the treatment order (i.e., starting with rhGH or placebo) was done through a permuted four-block designed by the physician, who was the only one not blinded for treatment allocation.

Drug: recombinant human growth hormone

Interventions

recombinant human growth hormone DRUG

RhGH treatment was started at 0.1 IU/kg once daily

Also known as: rhGH (Changchun Jinsai Pharmaceutical Co., Ltd, S20080011)

Group B

Saline DRUG

Saline was started at 0.1 IU/kg once daily as the placebo

Group A

Eligibility Criteria

• Age: 1 Year - 5 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• Patients diagnosed with PMS aged from 1years to 5 years

You may not qualify if:

• \- active or suspected tumour, intracranial hypertension, chronic kidney disease, acute proliferative or severe nonproliferative diabetic retinopathy, allergy to rhGH or severe comorbidity

Sponsors & Collaborators

• Affiliated Hospital of Jiangnan University: lead

Study Sites (1)

Affiliated Hospital of JiangNan University, Department of Pediatrics

Wuxi, Jiangsu, 226600, China

Location

MeSH Terms

Conditions

Telomeric 22q13 Monosomy Syndrome

Interventions

Growth Hormone; Long-Term Synaptic Depression; Sodium Chloride

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Neuronal Plasticity; Nervous System Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• YueYing Liu, Phd

  Affiliated Hospital of Jiangnan University

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 25, 2021
• First Posted: November 3, 2021
• Study Start: June 14, 2020
• Primary Completion: May 12, 2021
• Study Completion: June 8, 2021
• Last Updated: October 10, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)